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CTI-1601
CTI-1601 is a gene therapy that aims to restore motor neuron function in patients with amyotrophic lateral sclerosis (ALS).
CTI-1601 is a gene therapy that aims to restore motor neuron function in patients with amyotrophic lateral sclerosis (ALS). Used for Amyotrophic lateral sclerosis (ALS).
At a glance
| Generic name | CTI-1601 |
|---|---|
| Also known as | Nomlabofusp |
| Sponsor | Larimar Therapeutics, Inc. |
| Drug class | Gene therapy |
| Target | SOD1 gene |
| Modality | Biologic |
| Therapeutic area | Neurology |
| Phase | Phase 2 |
Mechanism of action
CTI-1601 uses a viral vector to deliver a healthy copy of the SOD1 gene to motor neurons, replacing the mutated gene that causes ALS. This approach has shown promise in preclinical studies, but its efficacy in humans remains to be determined.
Approved indications
- Amyotrophic lateral sclerosis (ALS)
Common side effects
- Injection site reactions
Key clinical trials
- An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia (PHASE2)
- A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia (PHASE1)
- A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia (PHASE2)
- Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia (PHASE1)
- Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- CTI-1601 CI brief — competitive landscape report
- CTI-1601 updates RSS · CI watch RSS
- Larimar Therapeutics, Inc. portfolio CI